COL7A1

Abeona Therapeutics Announces $50 Million Credit Facility

Retrieved on: 
Monday, January 8, 2024

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.

Key Points: 
  • CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.
  • The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.
  • The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.
  • “We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel,” said Joe Vazzano, Chief Financial Officer of Abeona.

Abeona Therapeutics Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA)

Retrieved on: 
Monday, November 27, 2023

CLEVELAND, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024. The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.

Key Points: 
  • Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024.
  • The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.
  • Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction.
  • The grant of the Priority Review status is an important prerequisite for Abeona’s eligibility for a Priority Review Voucher.

Abeona Therapeutics Reports Third Quarter 2023 Financial Results and Corporate Developments

Retrieved on: 
Monday, November 13, 2023

CLEVELAND, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the third quarter of 2023 and provided corporate updates.

Key Points: 
  • The FDA also agreed that retroviral vector manufactured at Abeona and Indiana University appear comparable based on the data that Abeona provided in its briefing book.
  • If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024.
  • Abeona estimates that its cash and cash equivalents, restricted cash and short-term investments as of September 30, 2023 are sufficient resources to fund operations into the fourth quarter of 2024.
  • Abeona Therapeutics will host a conference call and webcast today, November 13, 2023, at 8:30 a.m.

debra of America Raises $600,000 at Annual Benefit; Honors Trailblazers in Epidermolysis Bullosa Community

Retrieved on: 
Friday, November 10, 2023

Funds raised help advance EB research initiatives and directly support families and individuals living with EB.

Key Points: 
  • Funds raised help advance EB research initiatives and directly support families and individuals living with EB.
  • At the benefit, debra honored several EB trailblazers for their research and therapeutic contributions as well as individuals living with the disease.
  • Krystal Biotech was recognized for its work in developing VYJUVEK™ (beremagene geperpavec-svdt), the first FDA-approved treatment of Dystrophic Epidermolysis Bullosa (DEB).
  • Epidermolysis Bullosa is a rare genetic disorder characterized by fragile skin that easily blisters or forms painful sores from minor friction or trauma.

FDA Approves Groundbreaking New Product Utilizing Berkshire Sterile Excipient Gel to Treat 'Butterfly Children'

Retrieved on: 
Thursday, May 25, 2023

BSM supplies an excipient gel, a key component of this new and innovative therapy.

Key Points: 
  • BSM supplies an excipient gel, a key component of this new and innovative therapy.
  • VYJUVEK is the first and only FDA approved treatment for DEB and is designed to address the underlying genetic cause of the disease.
  • Berkshire Sterile Manufacturing manufactures an excipient gel that forms a component of the product, which is applied topically to open wounds on the skin.
  • “We are motivated to deliver these treatments to our clients and their patients, and that means meeting the most stringent quality standards throughout the manufacturing process.”

Option Care Health Selected by Krystal Biotech as Part of a Limited Distribution Specialty Pharmacy Network for VYJUVEK™

Retrieved on: 
Tuesday, May 23, 2023

BANNOCKBURN, Ill., May 23, 2023 (GLOBE NEWSWIRE) -- Option Care Health, Inc. (“Option Care Health”) (Nasdaq: OPCH) today announced that it was selected by Krystal Biotech, Inc. (“Krystal Biotech”) (Nasdaq: KRYS) as part of a limited distribution specialty pharmacy network for VYJUVEK™. VYJUVEK was approved by the U.S. Food & Drug Administration (FDA) on May 19, 2023 for the treatment of patients with dystrophic epidermolysis bullosa (DEB).

Key Points: 
  • BANNOCKBURN, Ill., May 23, 2023 (GLOBE NEWSWIRE) -- Option Care Health, Inc. (“Option Care Health”) (Nasdaq: OPCH) today announced that it was selected by Krystal Biotech, Inc. (“Krystal Biotech”) (Nasdaq: KRYS) as part of a limited distribution specialty pharmacy network for VYJUVEK™.
  • VYJUVEK was approved by the U.S. Food & Drug Administration (FDA) on May 19, 2023 for the treatment of patients with dystrophic epidermolysis bullosa (DEB).
  • Through the national footprint of over 95 specialty pharmacies strategically located across the U.S., Option Care Health will serve as the only specialty pharmacy provider for mixing and delivering prepared VYJUVEK gel administration syringes to professional healthcare and home settings.
  • Option Care Health will collaborate with Amedisys, Inc. on this effort, including for Amedisys nurses to deliver and administer VYJUVEK to patients.

Orsini Specialty Pharmacy Selected as part of the Limited Specialty Pharmacy Network For VYJUVEK™, the First Gene Therapy for the Treatment of Dystrophic Epidermolysis Bullosa

Retrieved on: 
Tuesday, May 23, 2023

ELK GROVE VILLAGE, Ill., May 23, 2023 /PRNewswire/ -- Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases and gene therapies, announced today that Krystal Biotech has selected it as part of the limited specialty pharmacy network for VYJUVEK™ (beremagene geperpavec-svdt), a topical gene therapy for the treatment of Dystrophic Epidermolysis Bullosa (DEB).

Key Points: 
  • ELK GROVE VILLAGE, Ill., May 23, 2023 /PRNewswire/ -- Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases and gene therapies, announced today that Krystal Biotech has selected it as part of the limited specialty pharmacy network for VYJUVEK™ (beremagene geperpavec-svdt), a topical gene therapy for the treatment of Dystrophic Epidermolysis Bullosa (DEB).
  • For more information about VYJUVEK, see the Full Prescribing Information or https://vyjuvek.com .
  • DEB is caused by mutations in the COL7A1 gene that affects the skin and mucosal tissues.
  • "Orsini Specialty Pharmacy is deeply committed to helping DEB patients and their families gain access to this revolutionary treatment and provide ongoing support in managing their condition."

FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa

Retrieved on: 
Friday, May 19, 2023

This gene encodes type VII collagen (COL7), which is an essential protein that helps strengthen and stabilize the outer and middle layers of the skin.

Key Points: 
  • This gene encodes type VII collagen (COL7), which is an essential protein that helps strengthen and stabilize the outer and middle layers of the skin.
  • When COL7A1 is deficient, skin layers can separate, causing painful and debilitating blisters and wounds.
  • DEB usually presents itself at birth and is divided into two major types depending on the inheritance pattern: recessive dystrophic epidermolysis bullosa (RDEB) and dominant dystrophic epidermolysis bullosa (DDEB).
  • Vyjuvek is a genetically modified (engineered in a laboratory) herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds.

New England Journal of Medicine Publishes Phase 3 Data on B-VEC in Patients with Dystrophic Epidermolysis Bullosa

Retrieved on: 
Wednesday, December 14, 2022

The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa,” will appear in the December 15, 2022 issue of the NEJM.

Key Points: 
  • The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa,” will appear in the December 15, 2022 issue of the NEJM.
  • The GEM-3 trial was a randomized, double-blind, intra-patient placebo-controlled multi-center trial designed to evaluate the efficacy and safety of B-VEC for the treatment of DEB.
  • In the trial, matched wounds receiving topical B-VEC or placebo were evaluated in 31 DEB patients over 26 weeks.
  • Krystal Biotech, Inc. (NASDAQ: KRYS) is a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases.

Abeona Therapeutics Announces Positive Topline Results with Both Co-Primary Endpoints Met in Pivotal Phase 3 VIITAL™ Study of EB-101

Retrieved on: 
Thursday, November 3, 2022

Abeona anticipates submitting results from this study, including further details with additional exploratory endpoints and the Week 12 results, for presentation at future medical meetings and for publication in a peer-reviewed journal.

Key Points: 
  • Abeona anticipates submitting results from this study, including further details with additional exploratory endpoints and the Week 12 results, for presentation at future medical meetings and for publication in a peer-reviewed journal.
  • The Week 12 results are similar to Week 24 results, achieving statistical significance for pain reduction and wound healing at all levels.
  • In the Phase 3 VIITAL study, EB-101 has been shown to both heal such large chronic wounds and significantly reduce pain.
  • Abeona Therapeutics will host a conference call and webcast on Thursday, November 3, 2022, at 8:30 a.m. EDT, to discuss the positive topline results from the VIITAL study.